Gene therapy for adults with Pompe disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests a one-time gene therapy (AB-1009) for adults with late-onset Pompe disease. It aims to see if the treatment is safe and improves muscle and breathing function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 65 years old.
- You have been on enzyme replacement therapy (like Lumizyme or Nexviazyme) for at least 6 months and had at least 10 infusions.
- Your lung function (FVC) is between 30% and 80% of what's expected for your age.
- You can walk at least 100 meters with or without a cane or walker.
- You do not need a breathing machine during the day, and you have not had gene therapy before.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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